A Letter From Henry, Emmanuel’s Father
I’m writing to you today to share the hope we have found through the Children’s Tumor Foundation for our son, Emmanuel, who lives with neurofibromatosis type 1 (NF1).
When our family first heard of his diagnosis, we were filled with questions and uncertainty. We didn’t know what to expect. As we learned more about NF, we realized that it could get worse with time, and that there was no cure.
Emmanuel had always dreamed of becoming a professional soccer player. But as a plexiform neurofibroma grew in his right hip, displacing organs and compressing his bone marrow, it became clear that his life would change.
The tumor causes him extreme pain, and he now uses a wheelchair much of the time. Walking is a daily struggle, and Emmanuel’s dream of playing soccer has become a distant memory. Our family traveled across several countries in search of treatment, facing robbery, discrimination, and homelessness.
But last year, Emmanuel started taking Koselugo, the first FDA-approved treatment for inoperable plexiform neurofibromas, thanks to research funded by the Children’s Tumor Foundation and supporters like you.
Since then, his tumor has softened, and though we know there will be challenges ahead, we are filled with so much hope!
Hope for the Future
The Children’s Tumor Foundation announced the FDA approval of selumetinib (now Koselugo) in 2020. Since then, it has been made available for thousands like my son who battle plexiform neurofibroma tumors.
But there is so much more to be done! That’s why CTF needs your help! Will you give today to help CTF give kids like Emmanuel access to care and find new solutions to fight NF?
Emmanuel and our entire family is full of resilience and gratitude for the CTF-initiated research that paved the way to drug approval—research made possible by you and others.
But this is just the beginning. Koselugo remains the only FDA-approved treatment for NF, and children and adults with NF need more options. We shouldn’t have to travel across countries to access care. With your help CTF we can fight every day for faster treatments and more breakthroughs that will improve the lives of all who live with NF.
Thanks to YOU and your gifts, CTF is the drug discovery engine for NF, and, together, we are achieving significant milestones:
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- An FDA-approved drug (Koselugo)
- Another treatment submitted for FDA approval this past July
- A topical MEK inhibitor gel for children and adults in a phase 3 clinical trial for
cutaneous neurofibromas - A platform clinical trial for brigatinib is in progress and proving to shrink NF2-SWN tumors
- A CTF investment in a biomarker study so that lives will not be lost to NF1-related malignant peripheral
nerve sheath tumors (MPNSTs)
And right now, over 60 clinical trials are underway, bringing us closer to more treatments for every type of NF. Among them is a clinical trial set to begin with a drug developed using AI—another CTF investment. None of this is possible without your commitment and your support!
But there is more work to do. We need your help to speed progress toward more treatments for all NF patients.
Will you make a year-end gift today?
Your donation will directly impact the lives of families like mine and accelerate the search for more treatments, more breakthroughs, and more hope for millions around the world living with NF.
The time is now. You can help bring new treatments to those who urgently need them most.
Make your year-end gift today at ctf.org/give
With deepest gratitude,
Henry, Emmanuel’s father
P.S. Your year-end gift today helps find new treatments for those living with NF like Emmanuel.
